Why Listening to Patients Matters in Rare Disease Research

Why Listening to Patients Matters in Rare Disease Research

Gene therapy isn’t one-size-fits-all: Patient associations are shaping trials, asking hard questions, and demanding realistic timelines for treatment delivery.

NORD scientific symposium aims to advance rare disease research

The National Organization for Rare Disorders (NORD) will host its first rare disease scientific symposium, in hopes of advancing rare disease research. The NORD Rare Disease Scientific Symposium will be held June 2-3 in Washington. Online registration is available. The symposium “offers a rare opportunity to bring together clinicians, researchers, and thought leaders from across specialties to share ideas, address challenges, and celebrate break…